What question did this study set out to answer?

To determine whether oral penicillamine therapy provides palliative or therapeutic benefits in boys diagnosed with Duchenne muscular dystrophy.

May 1, 1979Archives of Neurology

Treatment of Duchenne's Muscular Dystrophy With Penicillamine

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Authors

RRRobert I. RoelofsUniversity of Minnesota

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Implication

Randomized trial reveals no significant clinical benefit of penicillamine in boys with Duchenne muscular dystrophy, indicating the therapy does not alter disease progression.

Key Points

To determine whether oral penicillamine therapy provides palliative or therapeutic benefits in boys diagnosed with Duchenne muscular dystrophy.
Conducted a randomized trial in 11 boys with Duchenne muscular dystrophy assigned to placebo (group A, n = 6; mean age 86.7 ± 31.6 months) or 250 mg penicillamine three times daily (group B, n = 5; mean age 95.4 ± 43.4 months).
Administered 50 mg of daily pyridoxine to all participants across both groups throughout the intervention.
Assessed motor status across 14 to 16 months using timed functional tests, manometric measurements of muscle force, and manual muscle testing, evaluated via analysis of variance.
Analysis of variance showed no statistically significant differences in overall functional performance or muscle strength between the penicillamine and placebo groups after 14 to 16 months of treatment.

Cite This Study

Robert I. Roelofs (1979) studied this question.

synapsesocial.com/papers/6a8c2d6b95f0361a9ebf089d https://doi.org/10.1001/archneur.1979.00500410044005

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Implication

Randomized trial reveals no significant clinical benefit of penicillamine in boys with Duchenne muscular dystrophy, indicating the therapy does not alter disease progression.

Treatment of Duchenne's Muscular Dystrophy With Penicillamine

Discussion

Member takes

Implication

Key Points

Cite This Study

Treatment of Duchenne's Muscular Dystrophy With Penicillamine

Discussion

Member takes

Implication

Key Points

Cite This Study

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